Genome editing and the next generation of antiviral therapy.

Genome editing and the next generation of antiviral therapy.
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DOI:
10.1007/s00439-016-1686-2
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发表时间:
2016-09
期刊:
影响因子:
5.3
通讯作者:
Jerome, Keith R.
Jerome, Keith R.
中科院分区:
生物学2区
文献类型:
--
作者:
Stone, Daniel;Niyonzima, Nixon;Jerome, Keith R.

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工程化核酸内切酶如归巢核酸内切酶(HE)、锌指核酸酶(ZFN)、Tal效应物核酸酶(TALENS)和RNA引导的工程化核酸酶(RGEN或CRISPR/Cas9)可以靶向特异性DNA序列进行切割,并且被证明是基因编辑的有价值的工具。最近工程化的核酸内切酶已经显示出作为治疗遗传疾病和感染性病原体的治疗剂的巨大前景。在这篇综述中,我们讨论了最近使用HE,ZFN,TALEN和CRISPR/Cas9基因编辑平台作为抗病毒治疗的努力。我们还讨论了基因编辑抗病毒疗法在动物疾病模型中进行测试以及向人类应用过渡时所面临的障碍。
Engineered endonucleases such as homing endonucleases (HEs), zinc finger nucleases (ZFNs), Tal-effector nucleases (TALENS) and the RNA-guided engineered nucleases (RGENs or CRISPR/Cas9) can target specific DNA sequences for cleavage, and are proving to be valuable tools for gene editing. Recently engineered endonucleases have shown great promise as therapeutics for the treatment of genetic disease and infectious pathogens. In this review, we discuss recent efforts to use the HE, ZFN, TALEN and CRISPR/Cas9 gene-editing platforms as antiviral therapeutics. We also discuss the obstacles facing gene-editing antiviral therapeutics as they are tested in animal models of disease and transition towards human application.
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